General Study Notes Quiz
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Questions and Answers

Exonuclease chews back the 3' strand to generate 5' overhangs on each fragment.

False (B)

DNA ligase seals the gaps to generate one dsDNA molecule of the combined fragments.

False (B)

Humira is a monoclonal antibody used to treat breast cancer.

False (B)

Antibodies represent the largest category of protein therapeutics.

<p>True (A)</p> Signup and view all the answers

The discovery phase involves testing on humans, prior to learning about the drug.

<p>False (B)</p> Signup and view all the answers

The preclinical safety phase assesses the harm of different doses to small animals.

<p>True (A)</p> Signup and view all the answers

Phase I clinical trials primarily focus on testing the efficacy of the drug on disease phenotype.

<p>False (B)</p> Signup and view all the answers

Phase II clinical trials use test and control groups to determine the optimal dosing regime.

<p>True (A)</p> Signup and view all the answers

TALENs require the selection of a gRNA specific to the target location, which is why it's advantageous to use over CRISPR.

<p>False (B)</p> Signup and view all the answers

Introducing a point mutation into chromosomal DNA using CRISPR necessitates a Cas9 nuclease to facilitate DNA repair.

<p>True (A)</p> Signup and view all the answers

The PAM site must be in close proximity upstream of the gRNA binding site.

<p>False (B)</p> Signup and view all the answers

Donor DNA used in CRISPR-mediated point mutations must include flanking homologous regions to facilitate non-homologous end joining (NHEJ).

<p>False (B)</p> Signup and view all the answers

DCas9, a modified version of Cas9, retains its nuclease activity but has altered specificity.

<p>False (B)</p> Signup and view all the answers

DCas9 can be used to up-regulate gene expression by attaching a transcriptional repressor and directing it to a promoter.

<p>False (B)</p> Signup and view all the answers

Attaching a deaminase domain to dCas9 allows for site-specific mutations to be introduced without DNA cleavage.

<p>True (A)</p> Signup and view all the answers

Selection of a gRNA specific to a target location is required for TALENs.

<p>False (B)</p> Signup and view all the answers

Protein therapeutics can be produced by genetically modifying expression systems using recombinant DNA technology.

<p>True (A)</p> Signup and view all the answers

Recombinant DNA technology is a high cost approach for generating protein therapeutics.

<p>False (B)</p> Signup and view all the answers

Interferon is expressed in animal cells during bacterial infections.

<p>False (B)</p> Signup and view all the answers

Recombinant technology can be used to generate hybrid interferons from fragments of natural interferons to enhance therapeutic efficacy.

<p>True (A)</p> Signup and view all the answers

Half-life of a drug is the time it takes for the entire initial dose to be removed from the body.

<p>False (B)</p> Signup and view all the answers

Pegylation involves the attachment of polyethylene glycol (PEG) to molecules, which can decrease renal clearance and increase the half-life of therapeutics.

<p>True (A)</p> Signup and view all the answers

Dimerization of therapeutics always decreases the rate of degradation and increases the half-life.

<p>False (B)</p> Signup and view all the answers

Fusing an XTEN domain to a protein decreases its stability and solubility.

<p>False (B)</p> Signup and view all the answers

B-cells are HGPRT- and can proliferate through the salvage pathway.

<p>False (B)</p> Signup and view all the answers

Hybridomas can synthesize dGTP and dTTP due to their HGPRT+ status.

<p>True (A)</p> Signup and view all the answers

Non-human antibodies will not elicit an immune response when used in humans.

<p>False (B)</p> Signup and view all the answers

Chimeric antibodies contain the complete Fc region from humans and the entire Fv region from mice.

<p>False (B)</p> Signup and view all the answers

Complementarity determining regions (CDRs) are constant regions within the Fv region of an antibody.

<p>False (B)</p> Signup and view all the answers

Gibson assembly is a technique that can be used to create a 95% humanized antibody.

<p>True (A)</p> Signup and view all the answers

Hybridomas offer a long-term supply of monoclonal antibodies because they cannot be cryopreserved.

<p>False (B)</p> Signup and view all the answers

XenoMice are not related to the generation of humanized antibodies.

<p>False (B)</p> Signup and view all the answers

Onpattro is an RNAi drug specifically used for the treatment of hATTR.

<p>True (A)</p> Signup and view all the answers

Gammaretrovirus is a non-enveloped retrovirus with a large genome size.

<p>False (B)</p> Signup and view all the answers

Adenoviruses can integrate into the host genome.

<p>False (B)</p> Signup and view all the answers

The knob structure on adenovirus fiber proteins binds to surface proteins on the host cell membrane.

<p>True (A)</p> Signup and view all the answers

Adeno-associated virus (AAV) can replicate independently without a helper virus.

<p>False (B)</p> Signup and view all the answers

Lentivirus specifically targets dividing cells for stable integration.

<p>False (B)</p> Signup and view all the answers

HSV-1 integrates into host-cell chromosomes after infecting neurons.

<p>False (B)</p> Signup and view all the answers

Adenovirus replication occurs in the cytoplasm of the infected cell.

<p>False (B)</p> Signup and view all the answers

Herceptin is a monoclonal antibody that targets the HER2 receptor.

<p>True (A)</p> Signup and view all the answers

Abzymes are antibodies that act solely as inhibitors without catalytic activity.

<p>False (B)</p> Signup and view all the answers

Antisense oligonucleotides can be used to enhance mRNA expression.

<p>False (B)</p> Signup and view all the answers

RNAi refers to small interfering RNA molecules that promote degradation of target mRNAs.

<p>True (A)</p> Signup and view all the answers

Ribozymes are nucleic acids that can cleave other nucleic acids.

<p>True (A)</p> Signup and view all the answers

Monovalent antibodies have two Fab domains.

<p>False (B)</p> Signup and view all the answers

Aptamers are designed to bind to enzymes exclusively.

<p>False (B)</p> Signup and view all the answers

Antisense oligonucleotides must be resistant to degradation by cellular nucleases.

<p>True (A)</p> Signup and view all the answers

Flashcards

Exonuclease function

An enzyme that removes nucleotides from the ends of DNA strands.

3' overhangs

Unpaired nucleotides left at the end of a DNA strand after exonuclease action.

Complementary overhangs

Sticky ends of DNA that can pair due to base pairing rules.

DNA ligase

An enzyme that seals nicks in the sugar-phosphate backbone of DNA.

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Protein therapeutic

A protein used to treat diseases; often monoclonal antibodies.

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Largest category of protein therapeutics

Antibodies are the most common type of protein therapeutics.

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Phase I trials

Initial human testing focusing on drug safety and dosage.

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Phase III trials

Final human testing assessing effectiveness on disease outcomes.

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Recombinant DNA technology

A method to genetically modify organisms to produce proteins of interest.

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Advantages of recombinant protein production

Produces large quantities, is cost-effective, allows easy variant creation, and ensures reproducibility.

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Interferon therapeutics

Proteins produced to fight viral infections, often generated through recombinant technology.

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Hybrid interferon

A synthetic version of interferon created by combining fragments of natural interferons.

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Drug half-life

The time required for the amount of drug in the body to reduce to half its initial value.

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Pegylation

The process of attaching polyethylene glycol (PEG) to molecules to increase their half-life.

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Dimerization

The process of joining two identical molecules to enhance the stability and half-life of therapeutics.

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XTEN domain fusion

A technique to increase protein stability, solubility, and resistance to degradation, thus improving half-life.

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B-cells

B-cells are HGPRT+ and can proliferate via the salvage pathway but are not immortal.

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Myeloma cells

Myeloma cells are HGPRT- and cannot synthesize dGTP or dTTP, preventing proliferation.

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Hybridomas

Hybridomas are immortal cells, HGPRT+, that can synthesize dGTP and dTTP, allowing prolonged proliferation.

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Advantages of hybridomas

Hybridomas provide a consistent source of identical monoclonal antibodies and can be cryopreserved for long-term use.

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Drawback of non-human antibodies

Non-human antibodies trigger immune responses in humans due to species-specific Fc regions leading to destruction.

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Chimeric antibodies

Chimeric antibodies have a human Fc region combined with a non-human antigen-specific Fv region.

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Humanized antibodies

Humanized antibodies are 95% human, with human Fc and non-human CDRs for targeting antigens.

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Complementarity determining regions (CDRs)

CDRs are hypervariable regions within the Fv of antibodies that determine binding specificity for antigens.

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TALENs

Transcription Activator-Like Effector Nucleases used for gene editing by creating double-strand breaks in DNA.

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CRISPR advantages

CRISPR is simpler, cheaper, and faster than ZFNs and TALENs due to gRNA selection instead of full enzyme design.

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Cas9 nuclease

Enzyme in CRISPR that cuts DNA at specific locations, facilitating gene editing.

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Guide RNA (gRNA)

Molecule that directs Cas9 to target DNA sequence by base pairing.

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PAM site

Protospacer Adjacent Motif, a short DNA sequence necessary for Cas9 binding and activity.

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Donor DNA

Template DNA containing sequences needed for homology-directed repair after Cas9 cleavage.

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dCas9

Deactivated Cas9 that does not cut DNA but can still bind, allowing regulation of gene expression.

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FokI domain

Nuclease domain that can be added to dCas9 for targeted DNA cleavage at specific locations when paired with another gRNA.

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Herceptin

A monoclonal antibody targeting the HER2 receptor to prevent uncontrolled cell growth.

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Abzymes

Catalytic monoclonal antibodies that possess active sites for catalysis.

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Antisense Oligonucleotides

Short nucleic acids that bind mRNAs to inhibit their expression.

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Aptamers

Nucleic acids that bind to specific protein motifs or other molecules.

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Ribozymes

Nucleic acid enzymes capable of cleaving other nucleic acids.

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RNAi

Small interfering RNA that directs degradation of specific mRNAs.

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Characteristics of Antisense Oligonucleotides

Must be taken up by cells, target specific mRNAs, and resist degradation.

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Second/Third Generation Antisense Oligonucleotides

Advanced forms of antisense oligonucleotides with improved properties.

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siRNAs in Huntington's disease

siRNAs help degrade abnormal mRNAs with long CAG repeats to prevent protein aggregation in Huntington's disease.

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Onpattro

Onpattro is an RNAi drug used to treat hATTR by targeting faulty production of proteins.

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Gammaretrovirus

An enveloped retrovirus with a small genome, integrating into the host genome.

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Lentivirus

An enveloped retrovirus that can integrate into non-dividing cells, with a larger genome than gammaretroviruses.

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Adenovirus

A naked virus with a large dsDNA genome that infects various cells but does not integrate into the host genome.

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Adeno-associated virus (AAV)

A small, non-pathogenic virus that requires a helper virus for replication and can integrate into the host genome.

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HSV-1

An enveloped virus with a large dsDNA genome that primarily targets neurons and does not integrate into the host genome.

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Adenovirus infection process

The process where adenovirus binds to host cells, enters via a vesicle, and releases its DNA in the nucleus for replication.

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Study Notes

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Test your knowledge with this general study notes quiz. It's an open-ended evaluation designed to assess various topics without the constraints of specific texts or chapters. Perfect for reinforcing learning across diverse subjects.

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